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IRD vs RFL

Opus Genetics, Inc. Common Stock and Rafael Holdings, Inc. Class B Common Stock

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Opus Genetics, Inc. Common Stock

Opus Genetics, Inc., a clinical-stage ophthalmic biopharmaceutical company, focuses on developing and commercializing therapies for the treatment of inherited retinal diseases (IRDs). It develops OPGx-LCA5, an early-onset retinal degeneration that is in Phase 1/2 clinical trial for the treatment of LCA5-associated IRD; and OPGx-BEST1 to treat BEST1-associated retinal disease. The company also develops various pre-clinical IRD programs, including OPGx-RHO, a gene therapy that targets autosomal dominant retinitis pigmentosa caused by RHO mutations; OPGx-RDH12 that is designed to restore protein expression and halt functional deterioration in patients with retinal dystrophy caused by mutations in the retinal dehydrogenase gene; OPGx-MERTK, an AAV vector for the treatment of MERTK IRD; OPGx-NMNAT1, a gene augmentation therapy designed to halt functional deterioration in pediatric patients with retinal degenerative disease caused by mutations in the nicotinamide mononucleotide adenylyltransferase 1 gene; and OPGx-CNGB1, an AAV gene therapy for retinitis pigmentosa due to mutations in the CNGB1 gene. In addition, it develops Phentolamine Ophthalmic Solution for reversal of mydriasis, as well as for the treatment of presbyopia and dim light or night vision disturbances; and APX3330, a small-molecule inhibitor of Ref-1 for the treatment of diabetic retinopathy. The company was formerly known as Ocuphire Pharma, Inc. Opus Genetics, Inc. was founded in 2018 and is headquartered in Durham, North Carolina.

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Rafael Holdings, Inc. Class B Common Stock

Rafael Holdings, Inc. primarily engages in holding interests in clinical and early-stage pharmaceutical companies, and medical devices in the United States and Israel. It operates in three segments, Healthcare, Infusion Technology, and Real Estate. The company engages in the development and commercialization of therapies that exploit the metabolic differences between normal cells and cancer cells. Its lead drug candidate is Trappsol Cyclo, which is in phase 3 clinical trial being evaluated for the potential treatment of Niemann-Pick Disease Type C1 (NPC1), a rare, fatal and progressive genetic disorder; and CPI-613 (devimistat), a stable analog of normally transient, acylated catalytic intermediates of lipoate, which is in phase II of clinical trial. The company is also involved in developing Promitil, a molecule designed for the targeted delivery of mitomycin-C in a proprietary prodrug form, completed Phase 1A and 1B clinical studies targeting patients with advanced cancers. In addition, it engages in the development of surgical and procedural devices, including orthopedic arthroscopy instrument for Carpal Tunnel syndrome. Rafael Holdings, Inc. was incorporated in 2017 and is headquartered in Newark, New Jersey.

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